Targeted Therapy for Bone Cancer

As researchers have learned more about the molecular and genetic changes in cells that cause cancer, they have been able to develop newer drugs that specifically target some of these changes. These drugs, called targeted therapy drugs, work differently from standard chemotherapy (chemo) drugs and have different side effects. Targeted drugs are especially important in diseases like chordomas and other bone cancers, where chemo has not been very useful. They may prove to be a better treatment for these cancers. A lot of researchers are looking at how these drugs might be used to treat primary bone cancers.


Some chordomas have gene defects (mutations) that make proteins that signal the cells to grow. The drug imatinib (Gleevec®) is a targeted therapy drug that can block the signals from these genes. This can make some tumors stop growing or even shrink a little. Imatinib is used to treat chordomas that have spread or have come back after treatment. Imatinib has been used to treat chordomas for many years, but it isn’t approved by the FDA (Food and Drug Administration) to treat this type of cancer. It is approved to treat other cancers.

This drug is a pill, taken with food once a day. Common side effects are mild and can include diarrhea, nausea, muscle pain, and fatigue. These are generally mild. Some people taking the drug have itchy skin rashes. Fluid build-up around the eyes, feet, or belly can also be a problem.


Denosumab (Xgeva® or Prolia®) is a monoclonal antibody (a man-made version of an immune system protein) that binds to a protein called RANK ligand. RANK ligand normally tells cells called osteoclasts to break down bone, but when denosumab binds to it, that action is blocked. In patients with giant cell tumors of bone that have either come back after surgery or cannot be removed with surgery, denosumab can help shrink tumors for a while.

To treat giant cell tumors, this drug is injected under the skin (sub-q or SQ). Often, it can take months for the tumor to shrink.

Most side effects are mild and can include body aches, fatigue, and nausea. A rare but very distressing side effect of denosumab is damage to the jawbone called osteonecrosis of the jaw (ONJ). ONJ often starts as an open sore in the jaw that won’t heal. It can lead to loss of teeth and/or infections of the jaw bone. Doctors don’t know why this happens, but it can be triggered by having a tooth removed while taking the drug. The best treatment is also unclear, other than to stop denosumab. Maintaining good oral hygiene by flossing, brushing, making sure that dentures fit properly, and having regular dental check-ups may help prevent this. Most doctors recommend that patients have a dental check-up and have any tooth or jaw problems treated before they start taking this drug.


Interferons are not exactly targeted therapies. They're a family of substances naturally made by our immune system. Interferon-alpha is the type that may be used to treat giant cell tumors of the bone that have come back after treatment (recurred) or spread (metastasized).

This drug is most often given as a daily injection under the skin. It can also be injected into a muscle or vein.

Interferon can cause significant side effects. These include "flu-like" symptoms like muscle aches, bone pain, fever, headaches, fatigue, nausea, and vomiting. Patients taking this drug might have problems thinking and concentrating. Interferon can also lower blood cell counts. These effects continue as long as the drug is used, but can become easier to tolerate over time. They do improve after the drug is stopped. Still, some patients find it hard to deal with these side effects every day and may need to stop treatment because of them.

For more information about drugs that use the immune system, see Immunotherapy.

More information about targeted therapy

To learn more about how targeted drugs are used to treat cancer, see Targeted Cancer Therapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

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Last Revised: January 3, 2019